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LATEST NEWS
FDA Approves Fayuvi, the First Treatment for Sanfilippo Syndrome Type A
The FDA approved Ultragenyx's Fayuvi (UX111), an AAV9 gene therapy and the first treatment of any kind for Sanfilippo syndrome type A, often called childhood Alzheimer's. Here's how it works and what the data showed.
Sep 173 min read
FDA Grants Priority Review to What Could Be the World's First In Vivo CRISPR Therapy
The FDA accepted Intellia's application for lonvo-z in hereditary angioedema with priority review, setting a March 10, 2027 decision date. It could be the first CRISPR therapy that edits genes inside the body.
Sep 83 min read
FDA Places New Clinical Hold on Regenxbio's Hunter Syndrome Gene Therapy
The FDA placed a clinical hold on Regenxbio's RGX-121 for Hunter syndrome after MRIs found spinal nodules in 5 of 48 patients, derailing plans to resubmit. Here's the science and the timeline.
Aug 244 min read
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GENE TECH TIMES
The Future Has Arrived
FDA Approves Fayuvi, the First Treatment for Sanfilippo Syndrome Type A
The FDA approved Ultragenyx's Fayuvi (UX111), an AAV9 gene therapy and the first treatment of any kind for Sanfilippo syndrome type A, often called childhood Alzheimer's. Here's how it works and what the data showed.
Sep 17
FDA Places New Clinical Hold on Regenxbio's Hunter Syndrome Gene Therapy
The FDA placed a clinical hold on Regenxbio's RGX-121 for Hunter syndrome after MRIs found spinal nodules in 5 of 48 patients, derailing plans to resubmit. Here's the science and the timeline.
Aug 24
A Gene Therapy for Dravet Syndrome Cuts Seizures and Supports Development
At ASGCT 2026, Encoded Therapeutics shared one-year data for ETX101, an AAV9 gene regulation therapy for Dravet syndrome that cut seizures by about 76% at the top dose. A pivotal study is now underway.
May 13
Grace Science's GS-100 Earns RMAT Designation for NGLY1 Deficiency
Grace Science's GS-100, an AAV9 gene therapy for NGLY1 deficiency, earned FDA RMAT designation after treated children showed gains in motor and cognitive skills. Here's how it works and why it matters.
Apr 14
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